Glasdegib Medicine 12 June 2019 Extended indication Treatment of acute myeloid leukaemia (AML) in adult patients who are not candidates for standard induction chemotherapy. Therapeutic value No judgement Total cost € 45,675,000.00 Registration phase Registration application pending Product Active substance Glasdegib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication AML / MDS Extended indication Treatment of acute myeloid leukaemia (AML) in adult patients who are not candidates for standard induction chemotherapy. Current proprietary name Already available biosimilars / generics Proprietary name Daurismo Manufacturer Pfizer Portfolio holder Mechanism of action Other Route of administration Oral Therapeutical formulation Film-coated tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Glasdegib inhibits of the Hedgehog (Hh) signal transduction pathway via targeting Smoothened (SMO), a transmembrane receptor. Glasdegib inhibits SMO activitation and blocks Hedgehog signaling. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date May 2019 Expected Registration August 2020 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Registratie wordt verwacht in Q3 2020. Therapeutic value Current treatment options Lage dosering cytarabine. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration 100 mg References NCT03416179 Additional remarks Expected patient volume per year Patient volume < 261 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR Additional remarks De incidentie van AML bij personen van 75 jaar en ouder bedroeg 261 patiënten. Het aantal is afhankelijk van uiteindelijke indicatie. Een groot gedeelte van de patienten wordt in HOVON studies behandeld. Expected cost per patient per year Cost € < 175,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References FDA approves new blood cancer treatment from Pfizer, Reuters, November 21, 2018 Additional remarks In de VS is de lijstprijs gesteld op $16.925 per maand. De behandelduur is nog niet bekend maar voor een behandeling van een jaar zou dit neerkomen op $203.100 (ongeveer €175.000). De prijsstelling in de EU zal niet voor Q3 2020 beschikbaar zijn. Potential total cost per year Total cost € 45,675,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks